Today’s traditional drug development infrastructure is ill equipped to develop treatments that target the needs of individual patients. A learning environment for molecular diagnostics and biopharmaceuticals is needed. Introducing the third in a series of Anova White Papers on Creating a Learning Healthcare System, we discuss how an approach in partnership with an omics provider could help biopharmaceutical companies find patients with low prevalence omic targets, better educate healthcare providers on the meaning of precision medicine findings, tackle value and cost in reimbursement, and reach the goal of N=1 clinical trials.
Completion of the Human Genome Project issued forth the era of “omics” and the promise of precision medicine. Precision medicine promised to allow us to shift resources to prevention, help physicians make informed treatment decisions, reduce risk of adverse events and control healthcare cost. These promises have not come to fruition and only a relatively small number of targeted therapies have been approved for marketing with a molecular diagnostic test.
Co-development of a drug and diagnostic is difficult. It requires developers to have a strong theoretical understanding of their investigational products early in the development process. Added difficulty comes from the realization that complex disease may be defined by genetic heterogeneity. Today’s traditional drug development infrastructure is ill equipped to develop treatments that target the needs of individual patients. Transformation is needed to identify, approach and accrue patients with low prevalence targets into clinical trials.
Rather than launching studies at sites in the hope patients can be enrolled, properly matched patients must be provided the opportunity to participate in studies wherever they receive care. This could be done by materially expanding clinical trial opportunities at sites and establishing a stronger relationship between physicians and omics providers. A learning environment for molecular diagnostics and biopharmaceuticals is needed. This system would provide additional value to the medical community by educating healthcare providers on the biological mechanisms underlying response and resistance to molecularly targeted therapy, establish stronger connections between providers and omics providers and creating novel reimbursement strategies.
Successful use of biomarker testing also requires a clear regulatory pathway for personalized medicine diagnostics, consensus on common evidentiary standards for biomarker tests, an effective platform for collecting patient data on tests, treatments, outcomes, and a learning solution to improve patient care and outcomes. In the end, success may be found in N=1 studies and the ability of patients to participate in clinical trials wherever they receive care.
In the end, clinical trials will change from a focus on what works under most circumstances for the majority of patients with common chronic conditions to one where the individual patient is the sole unit of observation. N=1 studies will become increasingly common.
